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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.

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Title: Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Authors: Birch SM; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA., Lawlor MW; Medical College of Wisconsin, Milwaukee, WI 53226, USA., Conlon TJ; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA., Guo LJ; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA., Crudele JM; University of Washington, Seattle, WA 98109, USA., Hawkins EC; College of Veterinary Medicine, North Carolina State University, Raleigh, NC 27606, USA., Nghiem PP; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA., Ahn M; University of Nevada-Reno, Reno, NV 89557, USA., Meng H; Medical College of Wisconsin, Milwaukee, WI 53226, USA., Beatka MJ; Medical College of Wisconsin, Milwaukee, WI 53226, USA., Fickau BA; Medical College of Wisconsin, Milwaukee, WI 53226, USA., Prieto JC; University of North Carolina, Chapel Hill, NC 27599, USA., Styner MA; University of North Carolina, Chapel Hill, NC 27599, USA., Struharik MJ; Solid Biosciences Inc., Cambridge, MA 02142, USA., Shanks C; Solid Biosciences Inc., Cambridge, MA 02142, USA., Brown KJ; Solid Biosciences Inc., Cambridge, MA 02142, USA., Golebiowski D; Solid Biosciences Inc., Cambridge, MA 02142, USA., Bettis AK; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA., Balog-Alvarez CJ; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA., Clement N; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA., Coleman KE; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA., Corti M; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA., Pan X; School of Medicine, University of Missouri, Columbia, MO 65212, USA., Hauschka SD; University of Washington, Seattle, WA 98109, USA., Gonzalez JP; Solid Biosciences Inc., Cambridge, MA 02142, USA., Morris CA; Solid Biosciences Inc., Cambridge, MA 02142, USA., Schneider JS; Solid Biosciences Inc., Cambridge, MA 02142, USA., Duan D; School of Medicine, University of Missouri, Columbia, MO 65212, USA., Chamberlain JS; University of Washington, Seattle, WA 98109, USA., Byrne BJ; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA., Kornegay JN; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
Source: Science translational medicine [Sci Transl Med] 2023 Jan 04; Vol. 15 (677), pp. eabo1815. Date of Electronic Publication: 2023 Jan 04.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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